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Newborn Screening for X-Linked Adrenoleukodystrophy
Early diagnosis of males with X-linked adrenoleukodystrophy (X-ALD) is essential for preventing loss of life due to adrenal insufficiency and for timely therapy of the childhood cerebral form of X-ALD with hematopoietic cell transplantation. This article describes X-ALD, the current therapies, the h...
Autores principales: | Moser, Ann B., Jones, Richard O., Hubbard, Walter C., Tortorelli, Silvia, Orsini, Joseph J., Caggana, Michele, Vogel, Beth H., Raymond, Gerald V. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6715319/ https://www.ncbi.nlm.nih.gov/pubmed/31467997 http://dx.doi.org/10.3390/ijns2040015 |
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